Archive for May, 2011

Cytokinetics’ CK-2017357 – Expanding the muscle contractility pipeline to skeletal muscle disorders

Posted by lifetech on May 4th, 2011

Cytokinetics’ CK-2017357 is a small molecule that improves skeletal muscle contraction. CK-2017357 is now in phase II testing for two orphan neuromuscular diseases: amyotrophic lateral sclerosis (ALS) and myasthenia gravis (MG), as well as a third indication, peripheral vascular disease (PAD) with claudication. Phase IIa trial results reported last December showed evidence of clinical activity after a single dose on a range of exploratory clinical endpoints in ALS patients. The drug was safe and well tolerated, though milder adverse events were very common. The first clinical data in MG and PAD are expected by the end of the year, and additional ALS studies are already planned for the second half of 2011.

CK-2017357 is still early in development, but the data generated over the next 12 months will provide critical evidence as to the long-term prospects of the drug. Will activity observed in ALS translate into clinical benefit in larger long term studies? Will CK-2017357 find broad applicability across neuromuscular diseases? Can CK-2017357 improve claudication, a condition caused by ischemia rather than a motor neuron signaling deficiency? How significant are mild but common neurologic adverse events for tolerability and safety? How will Cytokinetics balance the market opportunity of a prevalent but non-fatal condition like PAD with rare but severe orphan diseases like ALS? We explore these and other issues in our most recent report on this young but promising drug.